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FDA-Approved Gene Therapies | Overview

Boston Children's Hospital offers two gene therapy treatments approved by the U.S. Food and Drug Administration (FDA) — Zolgensma for spinal muscular atrophy (SMA) and KYMRIAH CAR T-cell therapy for relapsed acute lymphoblastic leukemia (ALL).

Gene therapy for SMA (Zolgensma)

We are one of the first pediatric hospitals in the nation to offer a gene therapy treatment for SMA. Zolgensma, formerly AVXS-101, prevents further muscle degeneration by replacing the defective or missing gene (SMN1) that causes SMA. Learn more about SMA gene therapy.

CAR T-cell therapy for relapsed ALL

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center is a certified provider for the first FDA-approved CAR T-cell therapy called KYMRIAH for patients up to 25 years of age with relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL). Learn more about CAR T-cell therapy.